On April 20, 2026 Debiopharm (www.debiopharm.com), a privately-owned, Swiss-based biopharmaceutical company aiming to establish tomorrow’s standard of care to cure cancer and infectious diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the combination of its PKMYT1 inhibitor, lunresertib (Debio2513), and its WEE1 inhibitor, zedoresertib (Debio 0123).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The designation is for the treatment of adult patients with CCNE1 amplified, or a deleterious mutation in either FBXW7 or PPP2R1A, platinum-resistant/refractory ovarian cancer.

The FDA’s Fast Track program is designed to facilitate the development and expedite the review of new drugs intended to treat serious conditions and fill an unmet medical need. Programs granted Fast Track designation benefit from more frequent communication with the FDA and, if relevant criteria are met, may be eligible for Priority Review and Accelerated Approval of a New Drug Application (NDA).

Momentum Following AACR (Free AACR Whitepaper) Oral Presentation

This regulatory milestone follows the first clinical data disclosure from the MYTHIC Study (NCT04855656), a Phase I trial evaluating the lunresertib and zedoresertib combination in patients with advanced solid tumors harboring these specific genomic alterations. The data were featured yesterday in an oral presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting by Dr. Timothy A. Yap, Medical Oncologist and Physician-Scientist at The University of Texas MD Anderson Cancer Center, and Principal Investigator of the MYTHIC study.

"The FDA’s decision to grant Fast Track designation for this combination therapy validates our synthetic lethality approach to treating high-unmet-need cancers," said Esteban Rodrigo Imedio, Executive Medical Director, Oncology, Debiopharm. "Coming immediately after Dr. Yap’s presentation of the MYTHIC data at AACR (Free AACR Whitepaper), this designation highlights the potential of combining lunresertib and zedoresertib to provide a meaningful new clinical option for patients with biomarker-selected ovarian cancer who have exhausted platinum-based therapies."

Addressing Unmet Need in Ovarian Cancer

Platinum-resistant or refractory ovarian cancer remains one of the most challenging malignancies to treat, with limited effective options for patients whose tumors have developed resistance. By targeting the DNA Damage Response (DDR) pathway through the dual inhibition of PKMYT1 and WEE1, the lunresertib/zedoresertib combination aims to exploit specific genomic vulnerabilities (CCNE1, FBXW7, or PPP2R1A) to induce tumor cell death.

ABOUT DNA DAMAGE REPAIR (DDR)

When cells have damaged DNA, they must undergo a repair process known as DDR to survive. Cancer cells rely heavily on DDR as they divide and grow uncontrollably. Inhibition of DDR, particularly in combination with other anticancer agents, prevents cancer cells from repairing their DNA, ultimately activating a programmed cell death process. DDR inhibitors such as zedoresertib (Debio 0123), Debiopharm’s WEE1 inhibitor, are currently being investigated in clinical and preclinical studies.

ABOUT PKMYT1 INHIBITION

Lunresertib (Debio2513) is a first-in-class, oral PKMYT1 inhibitor designed to exploit specific genetic vulnerabilities in solid tumors, such as CCNE1 amplification. By targeting PKMYT1, the drug induces synthetic lethality, preventing cancer cells from repairing DNA damage and forcing them into programmed cell death. As the most advanced PKMYT1 inhibitor in clinical development, lunresertib has shown encouraging proof-of-concept results both as monotherapy and in combination therapies within the ongoing MYTHIC trial.

(Press release, Debiopharm, APR 20, 2026, View Source [SID1234664574])

On April 20, 2026 Flatiron Health reported the publication of the Validation of Accuracy for LLM/ML-Extracted Information and Data (VALID) Framework in the Journal of Clinical Oncology Clinical Cancer Informatics. The framework represents the first and most comprehensive, peer-reviewed approach to evaluating the quality and reliability of real-world data extracted by large language models (LLMs) and machine learning—setting a methodological benchmark for data integrity in oncology research.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

As large language models emerge as a tool for clinical data extraction from sources such as electronic health records, the industry faces a tradeoff—AI can unlock speed and scale, but it requires rigorous validation. Flatiron’s VALID Framework makes real-world data quality transparent and measurable, enabling evidence that meets the bar for high-stakes clinical decisions. Specifically, the framework applies a rigorous, three-pillar approach: variable-level performance metrics that benchmark LLM extraction against expert human abstraction; automated verification checks that systematically identify logical inconsistencies and implausibilities in data; and replication and benchmark analyses that confirm LLM-extracted results replicate established clinical findings.

"By publishing this framework transparently, we hope to contribute to raising the bar across the industry," said Nathan Hubbard, Chief Executive Officer of Flatiron Health. "Our commitment to data quality while applying LLMs responsibly and rigorously has enabled us to work at scale—with longitudinal records across millions of patients and over 1.5 billion data points—without compromising the rigor that has defined Flatiron for decades."

Flatiron’s LLM-extracted data builds on the highest-quality, human-abstracted real-world oncology data. By combining AI with expert human abstraction, Flatiron delivers gold-standard data quality at scale without trading off the clinical rigor that makes it fit for use in the highest-stakes decisions in cancer care and drug development. Every LLM-enabled dataset is subject to the VALID Framework, alongside long term clinical and scientific oversight to ensure data that captures complete patient journeys and validated outcomes.

"The VALID Framework, combined with our robust clinical and methodological expertise, gives us—and our customers—a clear basis for evaluating whether efficiency and accuracy go hand in hand, as well as confidence in clinical and strategic decisions made using real-world data," said Jonathan Kish, PhD, MPH, Vice President and Head of Research Sciences at Flatiron Health. "We’re investing deeply in the underlying work: data models, multimodal depth, and resolving complex edge cases to ensure that we’re not just extracting more data; we’re extracting better data at scale, so every decision is informed by intelligence you can trust."

Read the full publication: Estevez M, Singh N, Dyson L, et al. Ensuring Reliability of Curated EHR-Derived Data: The Validation of Accuracy for LLM/ML-Extracted Information and Data (VALID) Framework. JCO Clin Cancer Inform. 2026. View Source

(Press release, Flatiron Health, APR 20, 2026, View Source [SID1234664572])

On April 20, 2026 Deck Bio, a biotechnology company advancing multi-pMHC targeted T cell engagers for solid tumors, reported new preclinical data for its lead program, DBXO-1, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2026, which is being held April 17–22 in San Diego, California. The data were included in a poster titled "Preclinical characterization of DBXO-1, a multi-pMHC targeted bispecific T cell engager for major solid tumors."

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The data highlight Deck Bio’s strategy to overcome key limitations of T cell engagers in solid tumors, including low antigen density, tumor heterogeneity, and the scarcity of tumor-specific surface targets. DBXO-1 is designed to recognize multiple cancer-associated peptide–major histocompatibility complexes (pMHCs) using a single engineered T cell receptor (TCR)-based binder. By combining a proprietary TCR stabilization technology (dbTv) with a sequence-agnostic specificity profiling platform (dbSCOPE), DBXO-1 enables precise targeting of intracellular cancer antigens in a novel T cell engager format (dbTCE).

"The data presented at AACR (Free AACR Whitepaper) showcase the rationale and structural foundation that enables multi-pMHC targeting," said Johanna Kaufmann, Ph.D., Chief Scientific Officer of Deck Bio. "Leveraging our dbSCOPE technology, we prioritize specificity as a core design principle for DBXO-1, ensuring highly potent multi-target activity does not come at the expense of off-target toxicity."

Key Preclinical Findings from AACR (Free AACR Whitepaper) 2026

Engineered TCR-based binders demonstrated multi-target engagement with ~1–2 nM affinity across target pMHCs.
The structural recognition mode of DBXO-1 binders enables multi-pMHC targeting.
Deep specificity profiling using dbSCOPE (Deck Bio’s Sequence-agnostic Comprehensive Off-target Profiling Engine) to interrogate binding against 13,849 HLA-A*02:01-presented peptides from healthy tissues showed minimal off-target interactions and a specificity profile comparable to an approved pMHC-TCE.
Functional assays under stringent conditions demonstrated a favorable potency window, with greater than 1,000-fold EC50 selectivity for target versus off-target peptides.
DBXO-1 dbTCEs mediated T cell activation and potent target-dependent cytotoxicity, with EC50s comparable to an established pMHC-TCE.
DBXO-1 dbTCEs showed no alloreactivity with closely related HLA alleles, consistent with a highly specific recognition profile.
DBXO-1 dbTCEs, which contain the stabilized dbTv moiety, demonstrated extended half-life in human FcRn transgenic mice, with an estimated elimination half-life exceeding 7.5 days.
"These data support our thesis that a multi-target approach can expand the reach of T cell engagers in solid tumors while maintaining a high bar for specificity," said Jack Silberstein, Ph.D., Founder and Chief Executive Officer of Deck Bio. "We are advancing DBXO-1 with a disciplined focus on safety, durability of response, and the potential to treat broader patient populations."

Deck Bio is continuing preclinical development of DBXO-1, including additional safety and efficacy studies. The company’s initial clinical development is expected to focus on biomarker-selected patient populations across major solid tumor indications, including non-small cell lung and gastroesophageal cancers, representing an estimated addressable patient population of approximately 120,000 biomarker-positive patients in the first-line metastatic setting across the U.S., Europe, the U.K., and Australia.

(Press release, Deck Bio, APR 20, 2026, View Source [SID1234664571])

On April 20, 2026 Verismo Therapeutics, a clinical-stage CAR T cell therapy company pioneering a novel multichain KIR-CAR platform technology, reported the first clinical results from its ongoing Phase 1 STAR-101 clinical trial (NCT05568680) evaluating SynKIR-110 KIR-CAR living medicine in patients with advanced mesothelin-expressing solid tumors, including ovarian cancer, mesothelioma, and cholangiocarcinoma. These late-breaking data were presented during the Clinical Trial Plenary session 3 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2026 in San Diego, CA (Abstract CT 104).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are excited to share Verismo’s first presentation of clinical data evaluating our multi-chain SynKIR-110 KIR-CAR candidate, showing a positive safety profile, dose-dependent efficacy signals, and no dose-limiting toxicities," said Laura Johnson, Ph.D., Chief Operating Officer and Chief Scientific Officer of Verismo Therapeutics. "These data represent an important milestone for Verismo’s KIR-CAR platform, which is designed to combine the best of natural killer and T cells to fight cancer and may provide a more durable response than current single-chain CAR T therapies that are prone to exhaustion in difficult-to-treat solid tumor microenvironments."

Janos L. Tanyi, M.D., Ph.D., Principal Investigator for STAR-101 and Professor at the Perelman School of Medicine at the University of Pennsylvania (Penn), presented the data from nine patients treated across three dose-escalation cohorts. SynKIR-110 in dose level cohorts 1 through 3 showed a favorable safety profile, with no dose-limiting toxicities, no high-grade (Gr3+) cytokine release syndrome (CRS), and no immune effector cell-associated neurotoxicity syndrome (ICANS) events observed.

The initial data showed on-target biologic activity based on KIR-CAR T cell expansion and persistence in patients and serum cytokine changes after SynKIR-110 infusion. Anti-tumor activity was observed at increased dose levels, with 1 of 3 patients at dose level 3 achieving a partial response per RECIST criteria that was ongoing after 3+ months at the interim data cut-off for this dataset, which was September 2025. The trial remains ongoing, with continued enrollment to evaluate safety and determine the recommended Phase 2 dose.

"These data are significant because patients with mesothelin-expressing solid tumors continue to face significant and urgent needs for better treatments," said Dr. Tanyi, who is an associate professor of Obstetrics and Gynecology at Penn. "The safety profile we have observed, including low grade (Gr 1-2) CRS in only 3 of 9 patients, and no neurotoxicity, combined with early signs of clinical activity, support continued investigation of this novel approach in patients with solid tumors."

About SynKIR-110 KIR-CAR and the STAR-101 Clinical Trial

SynKIR-110 KIR-CAR is an investigational autologous engineered cell therapy developed using Verismo’s KIR-CAR platform. This approach utilizes a multi-chain, split-signaling architecture derived from natural killer cells, designed to drive long-term anti-tumor T cell function without T cell exhaustion. By keeping antigen recognition separate from T cell activation, the multi-chain architecture is intended to sustain T cell activity even in challenging solid-tumor microenvironments.

The STAR-101 clinical trial is a first-in-human, multicenter, open-label Phase 1 study in the U.S., designed to evaluate the safety, feasibility, and preliminary efficacy of SynKIR-110 in patients with advanced mesothelin-expressing solid tumors, including ovarian cancer, mesothelioma, and cholangiocarcinoma. These target indications are associated with poor prognosis and remain areas of high unmet medical need. The STAR-101 trial follows a dose-escalation design with an expansion cohort at the recommended Phase 2 dose. Patient recruitment and dose escalation in the trial is ongoing.

(Press release, Verismo Therapeutics, APR 20, 2026, View Source [SID1234664570])

On April 20, 2026 Independent biopharmaceutical company Specialised Therapeutics (ST) reported the approval of ZEPZELCA (lurbinectedin), in combination with atezolizumab (Tecentriq), as a new first-line maintenance treatment option for adult patients in Australia and Singapore, who have been diagnosed with extensive-stage small cell lung cancer (ES-SCLC), an aggressive form of lung cancer.[1],[2] This follows the recent decision by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) to issue a positive opinion recommending the approval of ZEPZELCA in Europe.[10]

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In combination with atezolizumab, ZEPZELCA has been approved for use in Australia and Singapore as a maintenance treatment for extensive-stage small cell lung cancer (ES-SCLC) in adult patients whose disease has not progressed after first-line induction therapy with atezolizumab, carboplatin and etoposide.[1],[2]

Both approvals for ZEPZELCA in ES-SCLC were obtained under Project Orbis — an initiative of the United States Food and Drug Administration’s (US FDA’s) Oncology Center of Excellence. These ZEPZELCA approvals represent the eighth time ST has successfully navigated the Project Orbis process since 2021.

Lung cancer remains a significant health challenge in the region, with an estimated 15,000 diagnoses and 9,000 deaths from the disease each year in Australia.[7],[8] In Singapore, lung cancer is the third most common cancer, with 9,732 new cases diagnosed between 2019-2023.[11] Small cell lung cancer (SCLC) is a highly aggressive cancer that has often already spread to other organs when first diagnosed.[6] It accounts for approximately 10-15% of all lung cancer cases and has a low 5-year survival rate of under 7%.[6]

Professor Nick Pavlakis, Medical Oncologist from Royal North Shore Hospital in Sydney, acknowledged the approval of the new combination therapy as an important step in helping to improve outcomes for patients with ES-SCLC.

"Most patients with small cell lung cancer typically present with extensive-stage disease at diagnosis, meaning they are often faced with experiencing high rates of relapse or recurrence, limited treatment options and a poor prognosis," said Professor Pavlakis.

"While outcomes for patients have improved over the past five years with the addition of immunotherapy to chemotherapy, there continues to be a significant clinical need to improve on first-line therapies used to treat extensive-stage small cell lung cancer to prolong disease control and enhance survival. Today’s announcement is welcome news for the medical and patient community."

ZEPZELCA is being made available in Australia, New Zealand, Singapore, Malaysia, Thailand and Vietnam by Specialised Therapeutics (ST), under exclusive license from European biopharmaceutical company PharmaMar, which has pioneered the development of marine-based oncology medicines.

ST Chief Executive Officer, Mr Carlo Montagner, said the approval of ZEPZELCA for ES-SCLC in the first-line setting would provide clinicians and patients with an important new therapeutic option for this difficult to treat cancer.

"We are delighted to have secured regulatory approvals for ZEPZELCA in combination with atezolizumab as a first-line maintenance therapy for adults diagnosed with ES-SCLC in Australia and Singapore," said Mr Montagner.

"Despite the improved efficacy of first-line therapies for ES-SCLC that have been available since 2019, survival outcomes remain suboptimal, with an estimated 40% of patients experiencing a relapse and requiring second-line treatment. The availability of additional first-line treatment options will enable eligible patients and their clinicians to find the therapy that is right for them, can help to improve their quality of life and importantly, give them more time to spend with their families and loved ones."

Chief Medical Officer for PharmaMar, Javier Jimenez, welcomed the regulatory approval for ZEPZELCA, saying: "We are very pleased that patients in Australia and Singapore can have access to this new therapy, as it marks an important milestone in the management of this disease, where a significant unmet medical need persists. Building on more than 40 years of commitment to research in diseases with limited treatment options, we will continue to collaborate with our partner in Australia to enable timely and equitable access to this new treatment."

ZEPZELCA belongs to a class of medicines known as alkylating agents, which work by damaging the DNA of cancer cells, helping to slow or stop their growth.[12] The regulatory approvals for ES-SCLC in Australia and Singapore were based on results from the Phase 3 IMforte clinical trial. The study enrolled 660 treatment-naïve patients with ES-SCLC across 13 countries in the induction phase. Following this, 483 patients who responded to induction treatment were randomised to receive either ZEPZELCA with atezolizumab (LU-AT) or atezolizumab monotherapy (AT) as first-line maintenance therapy.[13] The trial results supported the use of LU-AT as a new first-line maintenance treatment option over AT alone in patients with this aggressive cancer, with improvements recorded in both median progression free survival (PFS; 5.4 months vs. 2.1 months) and median overall survival (OS; 13.2 months vs.10.6 months).[13]

Significant variations in treatment-related adverse events (AEs) were noted in the two trial groups (83.5% in patients receiving LU-AT vs. 40% among patients receiving AT monotherapy).[13] Treatment with LU-AT was considered to be generally well tolerated, with no new or unexpected safety signals.[13] The most common adverse reactions (≥ 30%), including laboratory abnormalities, in patients who received ZEPZELCA with atezolizumab were decreased lymphocytes, decreased platelets, decreased haemoglobin, decreased leukocytes, decreased neutrophils, nausea, and fatigue/asthenia.

For further details on ZEPZELCA, contact your healthcare professional and refer to the approved Australian Consumer Medicine Information, available from the TGA’s website.*

PBS Information: ZEPZELCA is not listed on the Pharmaceutical Benefits Scheme (PBS).

(Press release, Specialised Therapeutics Australia, APR 20, 2026, View Source [SID1234664569])